A Placebo-controlled Study to Evaluate the Efficacy and Safety of Clenbuterol in Patients with Spinal and Bulbar Muscular Atrophy (SBMA)

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Azienda Ospedaliera di Padova

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

Trial duration

11 Apr 2024 → ongoing

Decision date (initial)

2024-09-16

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

No

Funding sources

AIFA

External identifiers

EU CT number

2024-516563-86-00

EudraCT number

2017-005103-27

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Efficacy

to assess the efficacy of chronic treatment with clenbuterol in patients with SBMA

Secondary objectives 2

1. the assessment of the persistence of the effect (if any) after prolonged treatment
2. the assessment of the tolerability of the assigned treatment in terms of treatment-emergent adverse effects

Conditions and MedDRA coding

Spinal and Bulbar Muscular Atrophy

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 5

1. males who have received a genetically confirmed diagnosis of SBMA (AR CAG repeat number >= 38);
2. aged between 18 and 75 (+364 days) years;
3. displaying one or more of the following clinical symptoms: muscle atrophy, limb weakness, bulbar palsy;
4. able to walk independently with or without a cane or other supporting device (all supporting devices are acceptable except on wheelchair);
5. providing a written informed consent.

Exclusion criteria 7

1. a documented cardiovascular disease precluding the use of beta2 agonists (in the judgment of the investigators);
2. glaucoma, severe prostatic hypertrophy, hyperthyroidism, pheochromocytoma, and other medical conditions that, in the judgment of the investigators, would expose the patient to undue risk of harm or prevent the patient from completing the study;
3. concomitant treatment with either beta-blockers or sympathomimetic drugs (If a beta-blockers concomitant medication is ongoing before the study inclusion, the patient can be enrolled if the beta-blocker is discontinued for 3 weeks prior to randomization visit);
4. inability to walk or walking only with the support of a caregiver;
5. use of beta2 agonists in the preceding 6 months;
6. participation to an interventional trial in the preceding 3 months;
7. neuromuscular disease other than SBMA.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. A responder to the assigned treatment (clenbuterol or placebo) will be defined as a subject with a 15% increase at V7 (48 weeks), compared to baseline, in the distance covered in six minutes at the 6MWT. The primary endpoint will be the percentage of responders in the two treatment arms.

Secondary endpoints 8

1. 6MWT during the 48-weeks treatment period (from V2 to V7) in the two treatment arms
2. SBMA-FRS total score during the 48-weeks treatment period (from V2 to V7) in the two treatment arms
3. AMAT total score during the 48-weeks treatment period (from V2 to V7) in the two treatment arms
4. FVC during the 48-weeks treatment period (from V2 to V7) in the two treatment arms
5. 6K total score during the 48-weeks treatment period (from V2 to V7) in the two treatment arms
6. serum creatinine levels during the 48-weeks treatment period (from V2 to V7) in the two treatment arms
7. ALSAQ-40 total score during the 48-weeks treatment period (from V2 to V7) in the two treatment arms
8. INQOL total score during the 48-weeks treatment period (from V2 to V7) in the two treatment arms

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Azienda Ospedaliera di Padova

Sponsor organisation

Azienda Ospedaliera di Padova

Address

Via Nicolo' Giustiniani 2

City

Padova

Postcode

35128

Country

Italy

Scientific contact point

Organisation

Azienda Ospedaliera di Padova

Contact name

Gianni Sorarù

Public contact point

Organisation

Azienda Ospedaliera di Padova

Contact name

Gianni Sorarù

Locations

1 EU/EEA country · 7 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 8 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

2 submissions — initial application plus substantial / non-substantial modifications